Vor Biopharma Inc
For the period ended Sep 30, 2024. Filed Nov 7, 2024.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2024 · Filed Nov 7, 2024
VOR$19.88+6.5%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-06-30; filed 2024-08-08.
0000950170-24-093858
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2024-09-30; filed 2024-11-07.
0000950170-24-123245
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 112 previous paragraphs and 118 current paragraphs.
88 unchanged; 23 reworded; 7 added; 1 removed; 0 previous and 0 current paragraphs rearranged.
Tables are not compared: 5 previous and 5 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (7)
Current report
Reliable engraftment, robust platelet recovery, and high CD33 editing efficiency with full myeloid chimerism at Day 28;
Current report
Shielding of the blood system, with maintained neutrophil and platelet counts across multiple Mylotarg doses of 0.5, 1, and 2 mg/m 2;
Current report
Broadened therapeutic index for Mylotarg with drug exposure represented by AUC which is related to efficacy, consistent with labeled Mylotarg doses, and with maximal concentrations, measured by Cmax and related to veno-occlusive disease, well below known toxic range; and
Current report
Early evidence suggesting patient benefit as measured by relapse-free survival when compared to published high-risk AML comparators, with only four patients relapsing, two of which occurred before Mylotarg treatment, and two occurring following Mylotarg, both with TP53 mutation.
Current report
We expect to provide our next clinical data update at the American Society of Hematology (ASH) 2024 annual meeting in December 2024.
Current report
In September 2024, we announced a new preclinical asset, VADC45, which has a number of potential opportunities in oncology, gene therapy, and autoimmune disorders. VADC45 is an ADC designed to target the CD45 protein. CD45 is a well-validated target for a wide variety of blood cancers with clinical proof of concept. The linker-payload used in VADC45 is also clinically validated. VADC45 has the potential to treat a number of diseases, including treatment of hematologic malignancies, as a targeted conditioning agent for gene therapies such as for sickle cell disease, holistic immune reset for autoimmune disorders, and for our approach of combining this asset with epitope modification of CD45 to shield healthy stem cells. We already have robust preclinical data for VADC45 and are progressing IND-enabling studies to enable future Phase 1 studies.
Current report
General and administrative expenses were $6.7 million for the three months ended September 30, 2024, compared to $7.7 million for the three months ended September 30, 2023. The decrease of $1.0 million was primarily due to a decrease in professional fees and stock-based compensation expense.
Removed paragraphs (1)
Previous report
General and administrative expenses were $15.2 million for the six months ended June 30, 2024, compared to $16.8 million for the six months ended June 30, 2023. The decrease of $1.6 million was primarily due to a decrease in consulting and legal expenses, partially offset by an increase in personnel costs.
Reworded paragraphs (23)
Previous wording
We are developing trem-cel, a shielded transplant, which we believe has the potential to transform the treatment for AML and other blood cancers. Trem-cel is created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target. We intend to develop trem-cel as a HCT product candidate to replace the standard of care in transplant settings. We are actively enrolling and treating patients in VBP101, our first-in-human Phase 1/2a trial of trem-cel in combination with Mylotarg. We released clinical data for this trial most recently in December 2023. The data showed that primary neutrophil engraftment occurred in all eight patients treated with trem-cel. Three out of three patients treated with Mylotarg experienced hematologic protection from deep cytopenias through repeat doses, suggesting that trem-cel transplants shielded patients’ healthy cells from the on-target toxicity typically seen with Mylotarg treatment. We expect to release additional engraftment and hematologic protection data and additional Mylotarg pharmacokinetic analyses in the second half of 2024. If successful, this trial will provide important validating evidence of the potential of trem-cel and our broader approach.
Current wording
We are developing trem-cel, a shielded transplant, which we believe has the potential to transform the treatment for AML and other blood cancers. Trem-cel is created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target. We intend to develop trem-cel as a HCT product candidate to replace the standard of care in transplant settings. We are actively enrolling and treating patients in VBP101, our first-in-human Phase 1/2a trial of trem-cel in combination with Mylotarg. We released clinical data on 18 patients treated in this trial in September 2024. The data demonstrated:
Previous wording
VCAR33 ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell product that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33 ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes. In January 2024 we dosed the first patient with VCAR33 ALLO in VBP301 and have treated multiple additional patients through the first half of 2024. We expect to report initial data in the second half of 2024.
Current wording
VCAR33 ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell product that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33 ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes. In January 2024 we dosed the first patient with VCAR33 ALLO in VBP301 and are continuing to dose patients in this study. In September 2024, we announced encouragingin vivo CAR-T expansion data from the first three patients treated, all at the lowest dose of 1 x 106 CAR+ cells/kg.
Previous wording
We operate an in-house clinical manufacturing facility in Cambridge, Massachusetts to support development of our shielded transplants and CAR-T therapeutic candidates for patients with blood cancers. While this facility is now operational, we continue to rely on third-party contract manufacturers for our required raw materials, manufacturing devices, active pharmaceutical ingredients and finished product for our research and clinical manufacturing. Since our inception in December 2015, we have devoted substantially all of our resources to raising capital, organizing and staffing our company, business and scientific planning, conducting discovery and research activities, acquiring or discovering product candidates, establishing and protecting our intellectual property portfolio, developing and progressing our product candidates and preparing for clinical trials, establishing arrangements with third parties for the manufacture of our product candidates and component materials, building out our internal clinical manufacturing facility, and providing general and administrative support for these operations. We do not have any product candidates approved for sale and have not generated any revenue from product sales. Through June 30, 2024, we funded our operations primarily through the sale of equity securities and debt financings and have received aggregate net proceeds from these transactions of approximately $464.3 million.
Current wording
We operate an in-house clinical manufacturing facility in Cambridge, Massachusetts to support development of our shielded transplants and CAR-T therapeutic candidates for patients with blood cancers. While this facility is now operational, we continue to rely on third-party contract manufacturers for our required raw materials, manufacturing devices, active pharmaceutical ingredients and finished product for our research and clinical manufacturing. Since our inception in December 2015, we have devoted substantially all of our resources to raising capital, organizing and staffing our company, business and scientific planning, conducting discovery and research activities, acquiring or discovering product candidates, establishing and protecting our intellectual property portfolio, developing and progressing our product candidates and preparing for clinical trials, establishing arrangements with third parties for the manufacture of our product candidates and component materials, building out our internal clinical manufacturing facility, and providing general and administrative support for these operations. We do not have any product candidates approved for sale and have not generated any revenue from product sales. Through September 30, 2024, we funded our operations primarily through the sale of equity securities and debt financings and have received aggregate net proceeds from these transactions of approximately $464.4 million.
Previous wording
We have incurred significant operating losses since inception, including net losses of $27.8 million and $58.6 million, respectively, for the three and six months ended June 30, 2024, and $117.9 million for the year ended December 31, 2023. As of June 30, 2024, we had an accumulated deficit of $398.7 million.
Current wording
We have incurred significant operating losses since inception, including net losses of $27.6 million and $86.2 million, respectively, for the three and nine months ended September 30, 2024, and $117.9 million for the year ended December 31, 2023. As of September 30, 2024, we had an accumulated deficit of $426.3 million.
Previous wording
As of June 30, 2024, we had cash, cash equivalents and marketable securities of $85.9 million. We expect that our cash, cash equivalents and marketable securities at June 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025.
Current wording
As of September 30, 2024, we had cash, cash equivalents and marketable securities of $62.8 million. We expect that our cash, cash equivalents and marketable securities at September 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025.
Previous wording
Interest income consists of interest income earned on our cash, cash equivalents and marketable securities held in financial institutions.
Current wording
Interest income consists of interest income earned on our cash, cash equivalents, restricted cash and marketable securities held in financial institutions.
Previous wording
Comparison of Three and Six Months Ended June 30, 2024 and 2023
Current wording
Comparison of Three and Nine Months Ended September 30, 2024 and 2023
Previous wording
Research and development expenses were $21.8 million for the three months ended June 30, 2024, compared to $23.9 million for the three months ended June 30, 2023. The decrease of $2.1 million was due to timing of purchases of manufacturing starting materials for our VCAR33 ALLO program and a decrease in preclinical expenses, offset in part by an increase in clinical trial costs to support our trem-cel and VCAR33 ALLO programs.
Current wording
Research and development expenses were $21.8 million for the three months ended September 30, 2024, compared to $27.6 million for the three months ended September 30, 2023. The decrease of $5.8 million was due to a decrease in license payments, preclinical expenses, and manufacturing starting materials, offset in part by an increase in clinical trial costs to support our trem-cel and VCAR33 ALLO programs.
Previous wording
Research and development expenses were $46.1 million for the six months ended June 30, 2024, compared to $45.8 million for the six months ended June 30, 2023. The increase of $0.3 million was primarily attributable to an increase in personnel-related costs driven by additional headcount to support our clinical and manufacturing activities and an increase in clinical trial costs to support our trem-cel and VCAR33 ALLO programs, offset in part by a decrease in preclinical expenses.
Current wording
Research and development expenses were $68.0 million for the nine months ended September 30, 2024, compared to $73.4 million for the nine months ended September 30, 2023. The decrease of $5.5 million was primarily due to a decrease in license payments, preclinical expenses, and stock-based compensation expense, partially offset by an increase in personnel-related costs driven by additional headcount to support our clinical and manufacturing activities and an increase in clinical trial costs to support our trem-cel and VCAR33 ALLO programs.
Previous wording
General and administrative expenses were $7.2 million for the three months ended June 30, 2024, compared to $8.3 million for the three months ended June 30, 2023. The decrease of $1.1 million was primarily due to a decrease in consulting and legal expenses, partially offset by an increase in personnel costs.
Current wording
General and administrative expenses were $21.9 million for the nine months ended September 30, 2024, compared to $24.5 million for the nine months ended September 30, 2023. The decrease of $2.6 million was primarily due to a decrease in professional fees and stock-based compensation expense, partially offset by an increase in personnel costs.
Previous wording
Other income decreased by $1.0 million during the three months ended June 30, 2024, compared to the three months ended June 30, 2023. Other income decreased by $1.5 million for the six months ended June 30, 2024, compared to the six months ended June 30, 2023. The decrease in other income in both periods was due to decreases in interest earned from our cash, cash equivalents and marketable securities as the Company held lower balances of cash, cash equivalents and marketable securities.
Current wording
Other income decreased by $1.2 million during the three months ended September 30, 2024, compared to the three months ended September 30, 2023. Other income decreased by $2.6 million for the nine months ended September 30, 2024, compared to the nine months ended September 30, 2023. The decrease in other income in both periods was due to decreases in interest earned from our cash, cash equivalents, restricted cash and marketable securities as the Company held lower balances of cash, cash equivalents and marketable securities.
Previous wording
Since our inception, we have not recognized any revenue and have incurred operating losses and negative cash flows from our operations. We have not yet commercialized any product and we do not expect to generate revenue from sales of any products for several years, if at all. We have funded our operations primarily through the sale of equity securities and have received aggregate net proceeds from financing transactions of approximately $464.3 million as of June 30, 2024.
Current wording
Since our inception, we have not recognized any revenue and have incurred operating losses and negative cash flows from our operations. We have not yet commercialized any product and we do not expect to generate revenue from sales of any products for several years, if at all. We have funded our operations primarily through the sale of equity securities and have received aggregate net proceeds from financing transactions of approximately $464.4 million as of September 30, 2024.
Previous wording
In order to fund our future operations, including our planned clinical trials, on March 14, 2022, we filed a universal shelf registration statement (the “Shelf Registration Statement”), to provide for aggregate offerings of up to $350.0 million of common stock, preferred stock, debt securities, warrants or any combination thereof. As of June 30, 2024, $274.5 million remains available under this Shelf Registration Statement, including $119.8 million reserved for at-the market offerings discussed below.
Current wording
In order to fund our future operations, including our planned clinical trials, on March 14, 2022, we filed a universal shelf registration statement (the “Shelf Registration Statement”), to provide for aggregate offerings of up to $350.0 million of common stock, preferred stock, debt securities, warrants or any combination thereof. As of September 30, 2024, $274.4 million remains available under this Shelf Registration Statement, including $119.7 million reserved for at-the market offerings discussed below.
Previous wording
In December 2022, we entered into a Sales Agreement with Stifel, Nicolaus & Company, Incorporated (“Stifel”) as the agent (the "Stifel ATM Facility"). Pursuant to the Stifel ATM Facility, we may offer and sell shares of common stock with an aggregate value of up to $125.0 million. We will pay Stifel a commission of up to 3.0% of the gross proceeds of any common stock sold through Stifel. We sold 139,462 shares of our common stock under the Stifel ATM Facility during the six months ended June 30, 2024 at a weighted-average price per share of $2.11 for aggregate net proceeds of $0.3 million, after deducting commissions. As of June 30, 2024, $119.8 million remained available to be sold under the Stifel ATM Facility.
Current wording
In December 2022, we entered into a Sales Agreement with Stifel, Nicolaus & Company, Incorporated (“Stifel”) as the agent (the "Stifel ATM Facility"). Pursuant to the Stifel ATM Facility, we may offer and sell shares of common stock with an aggregate value of up to $125.0 million. We will pay Stifel a commission of up to 3.0% of the gross proceeds of any common stock sold through Stifel. We sold 249,096 shares of our common stock under the Stifel ATM Facility during the nine months ended September 30, 2024 at a weighted-average price per share of $1.64 for aggregate net proceeds of $0.4 million, after deducting commissions. As of September 30, 2024, $119.7 million remained available to be sold under the Stifel ATM Facility.
Previous wording
As of June 30, 2024, we had cash, cash equivalents and marketable securities of $85.9 million. We will need to raise additional capital to fund our planned future operations. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that if we do obtain additional funding, that such funding will be obtainable on terms satisfactory to us.
Current wording
As of September 30, 2024, we had cash, cash equivalents and marketable securities of $62.8 million. We will need to raise additional capital to fund our planned future operations. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that if we do obtain additional funding, that such funding will be obtainable on terms satisfactory to us.
Previous wording
acquire or in-license products, product candidates, technologies;
Current wording
acquire or in-license products, product candidates, or technologies;
Previous wording
We did not have during the periods presented, and we do not currently have, any off-balance sheet arrangements that, have or are reasonably likely to have a material current or future effect on our financial condition, changes in financial condition, revenues or expenses, results of operations, liquidity, cash requirements or capital resources.
Current wording
We did not have during the periods presented, and we do not currently have, any off-balance sheet arrangements that have or are reasonably likely to have a material current or future effect on our financial condition, revenues or expenses, results of operations, liquidity, cash requirements or capital resources.
Previous wording
Net cash used in operating activities was $52.3 million for the six months ended June 30, 2024, reflecting a net loss of $58.6 million and net cash used of $2.7 million for operating assets and liabilities, which were offset by non-cash charges of $9.0 million. The non-cash charges primarily consisted of stock-based compensation expense of $5.9 million, non-cash lease expense of $2.5 million and depreciation expense of $1.8 million, offset by $1.1 million of non-cash interest earned on marketable securities.
Current wording
Net cash used in operating activities was $75.6 million for the nine months ended September 30, 2024, reflecting a net loss of $86.2 million and net cash used of $2.8 million for operating assets and liabilities, which were offset by non-cash charges of $13.4 million. The non-cash charges primarily consisted of stock-based compensation expense of $8.1 million, non-cash lease expense of $3.8 million and depreciation expense of $2.7 million, offset by $1.1 million of non-cash interest earned on marketable securities.
Previous wording
Net cash used in operating activities was $48.4 million for the six months ended June 30, 2023, reflecting a net loss of $58.4 million and net cash used of $0.2 million for operating assets and liabilities, which were offset by non-cash charges of $9.9 million. The non-cash charges primarily consisted of stock-based compensation expense of $8.3 million, non-cash lease expense of $2.3 million and depreciation expense of $1.7 million, offset by $2.4 million of non-cash interest earned on marketable securities.
Current wording
Net cash used in operating activities was $76.3 million for the nine months ended September 30, 2023, reflecting a net loss of $91.6 million, offset by non-cash adjustments to operating assets and liabilities of $1.4 million and non-cash charges of $13.9 million. The non-cash charges primarily consisted of stock-based compensation expense of $11.5 million, non-cash lease expense of $3.5 million and depreciation expense of $2.6 million, offset by $3.7 million of non-cash interest earned on marketable securities.
Previous wording
The $3.9 million increase in net cash used in operating activities for the six months ended June 30, 2024 compared to the six months ended June 30, 2023 was primarily due to a decrease in stock-based compensation expense and differences in the timing of payments for costs incurred during each respective period.
Current wording
The $0.7 million decrease in net cash used in operating activities for the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 was primarily due to a decrease in research and development expenses offset by differences in the timing of payments for costs incurred during each respective period.
Previous wording
Net cash provided by investing activities was $97.0 million for the six months ended June 30, 2024, which consisted of proceeds of $107.0 million from the maturity of marketable securities, offset by purchases of $9.9 million of marketable securities and $0.1 million of property and equipment. Net cash provided by investing activities was $20.1 million for the six months ended June 30, 2023, which consisted of purchases of $58.4 million of marketable securities and $0.5 million of property and equipment offset by proceeds of $79.0 million from the maturity of marketable securities.
Current wording
Net cash provided by investing activities was $96.9 million for the nine months ended September 30, 2024, which consisted of proceeds of $107.0 million from the maturity of marketable securities, offset by purchases of $9.9 million of marketable securities and $0.2 million of property and equipment. Net cash provided by investing activities was $60.7 million for the nine months ended September 30, 2023, which consisted of proceeds of $120.0 million from the maturity of marketable securities, offset by purchases of $58.4 million of marketable securities and $1.0 million of property and equipment.
Previous wording
Net cash provided by financing activities was $0.1 million for the six months ended June 30, 2024, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $0.2 million and proceeds from exercise of stock options and issuance of shares under the ESPP of $0.1 million, offset by $0.2 million of taxes paid related to net share settlement of equity awards. Net cash provided by financing activities was $2.7 million for the six months ended June 30, 2023, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $4.2 million, and proceeds from stock option exercises and purchases of common stock under the ESPP of $0.2 million offset by the payment of $0.7 million of issuance costs related to the underwritten public offering under our Shelf Registration Statement and concurrent private placement that closed in December 2022, and $0.9 million of taxes paid related to net share settlement of equity awards.
Current wording
Net cash provided by financing activities was $0.1 million for the nine months ended September 30, 2024, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $0.3 million and proceeds from exercise of stock options and issuance of shares under the ESPP of $0.1 million, offset by $0.3 million of taxes paid related to net share settlement of equity awards. Net cash provided by financing activities was $2.9 million for the nine months ended September 30, 2023, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $4.6 million, and proceeds from stock option exercises and purchases of common stock under the ESPP of $0.2 million, offset by the payment of $0.7 million of issuance costs related to the underwritten public offering under our Shelf Registration Statement and concurrent private placement that closed in December 2022, and $1.2 million of taxes paid related to net share settlement of equity awards.
Previous wording
Contractual obligations relate to future minimum lease payments for existing non-cancellable leases primarily relating to corporate office and laboratory real estate, with terms expiring through February 2030. During the six months ended June 30, 2024, there were no significant changes in contractual obligations and commitments from that described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations and Other Commitments” in our 2023 Annual Report.
Current wording
Contractual obligations relate to future minimum lease payments for existing non-cancellable leases primarily relating to corporate office and laboratory real estate, with terms expiring through February 2030. During the nine months ended September 30, 2024, there were no significant changes in contractual obligations and commitments from that described under the heading “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Contractual Obligations and Other Commitments” in our 2023 Annual Report.